Italfarmaco Provides Update on Ongoing Clinical Programs with Givinostat in Oral Presentation at XVIII International Conference on Duchenne and Becker Muscular Dystrophy

— Analyses of long-term study with Givinostat in Duchenne Muscular Dystrophy boys continue to show delayed disease progression —

— EPYDIS Phase 3 pivotal clinical trial continues as planned with preliminary results expected in second quarter of 2022 —

MILAN–(BUSINESS WIRE)–The Italfarmaco Group announced updates on its clinical development programs with Givinostat, its proprietary histone deacetylase (HDAC) inhibitor, in boys with Duchenne Muscular Dystrophy (DMD), at the virtual XVIII International Conference on Duchenne and Becker Muscular Dystrophy. In the presentation made on February 20, 2021, Paolo Bettica, MD, PhD, Chief Medical Officer at the Italfarmaco Group, provided evidence which continues to show a delay in disease progression in DMD boys aged 7-11 years at treatment start, supported by data on the 7-year follow-up period from the long-term study with Givinostat in addition to steroid treatment.

“We are very encouraged to see that the long-term study with Givinostat continues to show a benefit in boys with DMD, which further supports its potential as a treatment. Our Phase 3 pivotal clinical study is on track and continues as planned with 179 boys recruited, and we look forward to announcing the results in the second quarter of next year,” commented Dr. Paolo Bettica. “We have made significant progress despite the pandemic and instituted procedures to ensure the safety and well-being of all trial participants while being able to continuously provide access to the study drug, as well as to maintain the scientific validity and integrity of the trial. We are grateful to the clinical teams conducting the studies in all of the sites for continuing to work for the benefit of the participants.”

The on-going, long-term study (ClinicalTrials.gov: NCT03373968) with Givinostat in boys with DMD is an extension of the company’s Phase 2 trial (ClinicalTrials.gov: NCT01761292). The new follow-up analyses* after more than 7 years of treatment show that the mean age at loss of ambulation in DMD boys treated with Givinostat on top of corticosteroids is 16.0 years contrasted to the one in the Cooperative International Neuromuscular Research Group (CINRG) study, which was 13.4 years (Mc Donald et al., Lancet 2017). Moreover, the yearly rate of change of respiratory parameters such as Forced Vital Capacity % Predicted (FVC%) and Peak Expiratory Flow % predicted (PEF%) is -1.7% and 0% in contrast to the 4 to 6% yearly rate of decline in these parameters as demonstrated in natural history studies of a patient population comparable to the Givinostat cohort (Mayer et al., Paediatr Pulmonol 2015; Henricson et al., Muscle Nerve 2013; Abresch et al., Neuromuscul Disord 2013; Kinane et al., Journal of Neuromuscular Diseases 2018).

Prof. Eugenio Mercuri, Professor of Paediatric Neurology at the Catholic University, Rome, Italy, commented on these results, “Overall these results suggest a potential long-term beneficial effect of Givinostat in DMD boys. We look forward to the final results and remain hopeful that these results can support the registration of Givinostat for the treatment of DMD.”

In October 2020, the U.S. Food and Drug Administration (FDA) granted a Rare Pediatric Disease designation to Givinostat for the treatment of DMD, which allows an expedited review process for new treatment modalities. Earlier, the company also received an Orphan Drug designation and Fast Track designation for Givinostat from the FDA.

About Givinostat

Givinostat is an investigational drug discovered through Italfarmaco’s internal research and development efforts in collaboration with Lorenzo Puri (Santa Lucia Foundation, Rome) and his team, and partnerships with Telethon and Parent Project aps. It is being evaluated for safety and efficacy for the treatment of Duchenne- and Becker- Muscular Dystrophy. Givinostat inhibits histone deacetylases (HDACs). HDACs are enzymes that prevent gene translation by changing the three-dimensional folding of DNA in the cell. Studies show that Duchenne patients have higher than normal HDAC levels, which may prevent muscle regeneration, and also trigger inflammation. In the company’s clinical study in DMD boys aged seven to less than 11 years, Givinostat was observed to slow disease progression, significantly increase muscle mass and reduce the amount of fibrotic tissue. Givinostat treatment also significantly reduced muscle tissue necrosis and fatty replacement, two additional parameters related to disease progression (Bettica et al., Neuromuscular Disorder 2016).

About Italfarmaco Group

Italfarmaco is a specialty pharmaceutical company engaged in the discovery, development, manufacturing and marketing of branded prescription and nonprescription products in more than 60 countries on 5 continents. Italfarmaco’s research and development expertise is best demonstrated through its HDAC inhibitor development programs, addressing new therapeutic treatments of specialty and rare diseases. Through both marketed drugs and compounds in development, Italfarmaco is dedicated to serving patients whose needs remain largely unmet.

*The results of these analyses are not published yet.